BPC December 13 update

​Amarin AMRN wins early FDA Approval; Intercept ICPT Advisory Committee meeting set - PDUFA to be delayed

Price and Volume Movers

Amarin Corporation plc (NASDAQ: AMRN) received approval from the FDA for the use of Vascepa as an secondary therapy to reduce the risk of cardiovascular events among adults with elevated triglyceride levels (a type of fat in the blood). Patients must also have either established cardiovascular disease or diabetes and two or more additional risk factors for cardiovascular disease. The early decision comes ahead of the assigned PDUFA date of December 28, 2019. Shares are currently trading up 5% after hours to $25.30.

Athenex, Inc. (Nasdaq: ATNX) announced further data from its Phase 3 clinical trial of oral paclitaxel and encequidar compared to IV paclitaxel in the treatment of metastatic breast cancer. Overall survival was recorded at 27.9 months vs 16.9 months for oral paclitaxel and encequidar and IV paclitaxel (p=0.035), respectively. Confirmed response rates were 40.4% for oral paclitaxel vs 25.6% for IV paclitaxel. The oral paclitaxel group also showed a lower incidence of alopecia and a lower incidence and severity of neuropathic AEs compared to IV paclitaxel (17% versus 57%). Progression free survival data favoured oral paclitaxel and encequidar at 9.3 months vs 8.3 months but were not significantly significant (p=0.077). Shares closed down 14% to $14.55.

Sarepta Therapeutics, Inc. (NASDAQ: SRPT) shares closed up 31% to $132.05 after surprising markets late-Thursday with news that the FDA approved Vyondys 53 (golodirsen) injection to treat Duchenne muscular dystrophy (DMD) patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping.

Aquestive Therapeutics, Inc. (NASDAQ:AQST) shares closed down 21% to $5.31 following the pricing of its public offering of 7m shares at a price of $5 per share for gross proceeds of $35m.

Intercept Pharmaceuticals, Inc. (Nasdaq:ICPT) announced the FDA has announced a tentative date of April 22, 2020 for their Advisory Committee meeting to discuss the New Drug Application (NDA) of obeticholic acid (OCA) for the treatment of fibrosis due to nonalcoholic steatohepatitis (NASH). As a result its PDUFA date of March 26, 2020 will be extended with a revised date to be confirmed.

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Major price movers (stocks priced > $1.00, volume > 50k):

Refer to the BioPharmCatalyst Biotech Stocks page for Top 10 Gainers/Losers/Unusual Volume

ADVANCERS:

NantKwest, Inc. (NK): $2.88; +31%.

Adial Pharmaceuticals, Inc. (ADIL): $2.20; +31%.

scPharmaceuticals Inc. (SCPH): $2.06; +21%.

BiondVax Pharmaceuticals Ltd. (BVXV): $8.29; +20%.

Entera Bio Ltd. (ENTX): $2.24; +19%.

DECLINERS:

OncoSec Medical Incorporated (ONCS): $2.02; -16%.

Anixa Biosciences, Inc. (ANIX): $3.26; -15%.

Fulcrum Therapeutics, Inc. (FULC): $11.76; -13%.

PhaseBio Pharmaceuticals, Inc. (PHAS): $3.37; -13%.

Synlogic, Inc. (SYBX): $2.38; -12%.

Pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst Market Cap

ALEC – Alector Inc.
AL002
Alzheimer’s disease

Phase 1b Phase 1b proof of mechanism data due in 2020.
$2.2 billion

ALEC – Alector Inc.
AL001
Frontotemporal Dementia

Phase 2 Phase 2 data due 1H 2020.
$2.2 billion

ALEC – Alector Inc.
AL003
Alzheimer’s disease

Phase 1 Phase 1b proof of mechanism data due in 2020.
$2.2 billion

AMRN – Amarin Corporation plc
Vascepa
High Triglycerides With Mixed Dyslipidemia

Approved FDA Approval announced December 13, 2019.
$6.3 billion

ATNX – Athenex Inc.
Oraxol
Metastatic breast cancer

Phase 3 Phase 3 data noted PFS (p=0.077) 9.3 months vs 8.3 months. OS (p=0.035) 27.9 months vs 16.9 months. ORR 40.4% vs 25.6%. - December 13, 2019.
$1 billion

BIIB – Biogen Inc.
BIIB092 - Anti Tau
Progressive supranuclear palsy (PSP)

Phase 2 Phase 2 trial did not meet primary endpoint - December 13, 2019.
$56.7 billion

BLRX – BioLineRx Ltd.
BL-8040 in combination with KEYTRUDA - COMBAT trial
Pancreatic cancer

Phase 2 Phase 2 PFS and OS data due mid-2020.
$23.8 million

HZNP – Horizon Therapeutics Public Limited Company
Teprotumumab
Thyroid Eye Disease (TED)

Approved FDA Approval announced January 21, 2020.
$6.5 billion

ICPT – Intercept Pharmaceuticals Inc.
Ocaliva (Obeticholic acid (OCA)) - REGENERATE
Adult nonalcoholic steatohepatitis (NASH) patients.

PDUFA priority review Advisory Committee meeting April 22, 2020. PDUFA date originally set for March 26, 2020 extended to June 26, 2020.
$3 billion

IPHA – Innate Pharma S.A.
IPH4102 - TELLOMAK
Mycosis Fungoides and Peripheral T-cell Lymphoma

Phase 2 Phase 2 outcome of the first stage of MF and PTCL cohorts due 2H 2020 with first efficacy data due early 2021. Phase 2 trial placed on partial clinical hold - noted January 9, 2020.
$581.2 million

LLY – Eli Lilly and Company
Empagliflozin
Heart failure with reduced ejection fraction (HFrEF)

Phase 3 Phase 3 data did not meet primary endpoint - December 13, 2019.
$132.1 billion

LLY – Eli Lilly and Company
Empagliflozin
Heart failure with preserved ejection fraction (HFpEF)

Phase 3 Phase 3 data did not meet primary endpoint - December 13, 2019.
$132.1 billion

MRTX – Mirati Therapeutics Inc.
Sitravatinib and tislelizumab
Ovarian cancer

Phase 1 Phase 1 data noted ORR 23.5% - December 13, 2019.
$3.8 billion

ONCS – OncoSec Medical Incorporated
TAVO + KEYTRUDA KEYNOTE-890
Triple negative breast cancer (TNBC)

Phase 2 Phase 2 interim data at SABCS December 12, 2019 noted ORR 28.5%. Full data due 2020.
$36.6 million